Karin Hoelzer’s Post

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Senior Director, Patient Advocacy at BIO

#developing #rarediseasetherapies is hard. Developing them for #rarepediatricdiseases is even harder. How do we bring more therapies to market for #children? How do we develop the right FDA strategies -and what impact will the new (ish) #FDA guidances have? How do we ensure we have the right incentives including pediatric rare disease priority review vouchers #PRVs- and how do #BPCA and #PREA work together as ‚carrot‘ and ‚stick‘? Thank you Laurie Conklin, MD , Christine Lee-Flemming, PhD, PharmD, Dionna Green and Thomas Miller for a fascinating and thought-provoking conversation at #Bio2024! My key take-away: we want ( need!) to include children earlier, but we need to get this right and have a strong scientific justification to inform the study design, and to get there we need the sophistication to leverage all the tools in the tool belt including #clinicaltrial simulation studies and model-informed trial design. A brave new world is upon us - if we can make the leap successfully!

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Thomas Miller

Life Science Executive, Board Director & Strategic Advisor

6mo

Could not agree more Karin - thanks for attending!

Jessica Fabre

Keynote Speaker - Disability Advocate - Author-Realtor- Executive Administrative Manager

5mo

Absolutely agree.

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